BAX 855 Previously Untreated Patient (PUP)



Status:Recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any - 5
Updated:4/25/2018
Start Date:November 12, 2015
End Date:June 29, 2023
Contact:Celine Kefurt
Email:celine.kefurt@baxalta.com
Phone:+431201002476205

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Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity, and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) and Minimally Treated Patients (MTPs) < 6 Years With Severe Hemophilia A (FVIII < 1%)

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of
PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of
age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or
plasma transfusion.


Inclusion Criteria

1. Participant is < 6 years old at the time of screening

2. Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or
plasma transfusion at any time prior to screening

3. Participant has severe hemophilia A (Factor VIII (FVIII) < 1%) as determined by the
central laboratory, or a historical FVIII level < 1% as determined at any local
laboratory, optionally supported by an additional FVIII gene mutation consistent with
severe hemophilia A

4. Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by
the central laboratory at screening

5. Parent or legally authorized representative is willing and able to comply with the
requirements of the protocol

Additional inclusion criteria for Part B (immune tolerance induction (ITI))

1. Parent or legal representative has/have voluntarily provided signed informed consent
for ITI portion

2. Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU))
or has a positive confirmed low titer inhibitor (≥ 0.6 BU) as determined by the
central laboratory based on a second repeat blood sample with

1. poorly controlled bleeding despite increased BAX 855 doses, or

2. requires bypassing agents to treat bleeding

Exclusion Criteria

1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
modification of the Bethesda assay) as confirmed by central laboratory at screening

2. Participant has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
modification of the Bethesda assay or the Bethesda assay) at any time prior to
screening

3. Participant has been diagnosed with an inherited or acquired hemostatic defect other
than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease)

4. Participant has been previously treated with any type of FVIII concentrate other than
ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for ≥ 3
EDs at any time prior to screening

5. Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment
and during the screening period, until the baseline infusion.

6. The participant's weight is anticipated to be < 5 kg at the baseline visit

7. Participant's platelet count is < 100,000/mL

8. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene
glycol (PEG) or Tween 80

9. Participant has severe chronic hepatic dysfunction [eg, > 5 times upper limit of
normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a
documented international normalized ratio (INR) > 1.5] in his medical history or at
the time of screening

10. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit
of normal)

11. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to
study participation or is scheduled to use such drugs during study participation

12. Participant is scheduled to receive during the course of the study a systemic
immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to
hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral
chemotherapy

13. Participant has participated in another clinical study involving an investigational
product (IP) or investigational device within 30 days prior to enrollment or is
scheduled to participate in another clinical study involving an IP or investigational
device during the course of this study

14. Parent or legally authorized representative has a medical, psychiatric, or cognitive
illness or recreational drug/alcohol use that, in the opinion of the investigator,
would affect participant safety or compliance

15. Parent, legally authorized representative or participant are a member of the team
conducting this study or is in a dependent relationship with one of the study team
members. Dependent relationships include close relatives (ie, children,
partner/spouse, siblings, parents) as well as employees of the investigator or site
personnel conducting the study.

Additional exclusion criteria for Part B (ITI)

1. Spontaneous disappearance of the inhibitor prior to ITI

2. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and
determined at the central laboratory

3. Inability or unwillingness to comply with the protocol
We found this trial at
14
sites
201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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100 N Mario Capecchi Dr
Salt Lake City, Utah 84132
(801) 662-1000
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200 Hawthorne Ln
Charlotte, North Carolina 28204
(704) 384-4000
Presbyterian Hospital At Novant Health Presbyterian Medical Center, we are welcoming a new era in...
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Chicago, Illinois 60611
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Cincinnati, Ohio 45229
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Cleveland, Ohio 44106
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3450 Hull Road
Gainesville, Florida 32610
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Hershey, Pennsylvania 17033
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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New York, New York 10021
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Peoria, Illinois 61615
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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949 University Avenue
Sacramento, California 95817
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Vienna,
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